Gene therapy aims to halt blindness from rare eye disease

NCT ID NCT03293524

First seen Apr 30, 2026 · Last updated May 16, 2026 · Updated 4 times

Summary

This study tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that causes sudden vision loss. Participants received injections into both eyes and were followed for up to 5 years. The goal was to see if the treatment could improve or stabilize eyesight compared to a placebo.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • CHNO Les Quinze Vingts

    Paris, Paris, 75012, France

  • Department of Ophthalmology, Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107, United States

  • Doheny Eye Center UCLA Pasadena

    Pasadena, California, 91105, United States

  • Emory Healthcare - The Emory Clinic

    Atlanta, Georgia, 30322, United States

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034, Spain

  • IRCCS Istituto delle Scienze Neurologiche di Bologna (ISNB) Clinica Neurologica

    Bologna, 40139, Italy

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    London, Greater London, EC1V 2PD, United Kingdom

  • Taipei Veterans General Hospital

    Taipei, 11217, Taiwan

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • University of Colorado Health Eye Center

    Aurora, Colorado, 80045, United States

  • Vanderbilt Eye Institute

    Nashville, Tennessee, 37232, United States

Conditions

Explore the condition pages connected to this study.